CA3089331A1 - Modulation de la transcription chez des animaux a l'aide de systemes crispr/cas - Google Patents

Modulation de la transcription chez des animaux a l'aide de systemes crispr/cas Download PDF

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Publication number
CA3089331A1
CA3089331A1 CA3089331A CA3089331A CA3089331A1 CA 3089331 A1 CA3089331 A1 CA 3089331A1 CA 3089331 A CA3089331 A CA 3089331A CA 3089331 A CA3089331 A CA 3089331A CA 3089331 A1 CA3089331 A1 CA 3089331A1
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sequence
human animal
protein
seq
target
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CA3089331A
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Charleen Hunt
Suzanne HARTFORD
Guochun Gong
Brian Zambrowicz
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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Publication of CA3089331A1 publication Critical patent/CA3089331A1/fr
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • A—HUMAN NECESSITIES
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    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
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    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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Abstract

Il est décrit des cellules animales non humaines et des animaux non humains comprenant une cassette d'expression qui comprend un acide nucléique codant une protéine de Cas associée à une courte répétition palindromique groupée et régulièrement espacée comprenant une protéine de Cas de nucléase inactive fusionnée à au moins un domaine d'activation transcriptionnelle; et un acide nucléique codant une protéine adaptateur chimérique qui comprend un adaptateur fusionné à au moins un domaine d'activation transcriptionnelle, la protéine adaptateur étant une protéine qui reconnaît spécifiquement une séquence d'acide ribonucléique distincte, et qui se lie à cette dernière, ladite séquence étant un élément se liant à un adaptateur. La cassette d'expression est intégrée dans un promoteur Rosa26, et elle est liée fonctionnellement à un promoteur Rosa26 de Rosa26. Il est décrit des procédés d'utilisation de tels animaux non humains pour augmenter l'expression de gènes cibles in vivo et pour évaluer des systèmes médiateurs d'activation synergiques de courte répétition palindromique groupée et régulièrement espacée et de Cas quant à la capacité d'augmenter l'expression de gènes cibles in vivo.
CA3089331A 2018-03-19 2019-03-19 Modulation de la transcription chez des animaux a l'aide de systemes crispr/cas Pending CA3089331A1 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862644961P 2018-03-19 2018-03-19
US62/644,961 2018-03-19
PCT/US2019/023009 WO2019183123A1 (fr) 2018-03-19 2019-03-19 Modulation de la transcription chez des animaux à l'aide de systèmes crispr/cas

Publications (1)

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CA3089331A1 true CA3089331A1 (fr) 2019-09-26

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CA3089331A Pending CA3089331A1 (fr) 2018-03-19 2019-03-19 Modulation de la transcription chez des animaux a l'aide de systemes crispr/cas

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Country Link
US (2) US11519004B2 (fr)
EP (1) EP3592140A1 (fr)
JP (1) JP7334178B2 (fr)
KR (2) KR20240038811A (fr)
CN (2) CN116349651A (fr)
AU (2) AU2019239880B2 (fr)
CA (1) CA3089331A1 (fr)
WO (1) WO2019183123A1 (fr)

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EP3585162B1 (fr) 2017-09-29 2023-08-30 Regeneron Pharmaceuticals, Inc. Rongeurs comprenant un locus ttr humanisé et procédés d'utilisation
AU2019239880B2 (en) 2018-03-19 2023-11-30 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using CRISPR/Cas systems
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EP3813522A1 (fr) 2019-06-14 2021-05-05 Regeneron Pharmaceuticals, Inc. Modèles de tauopathie
WO2021108363A1 (fr) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Régulation à la hausse médiée par crispr/cas d'un allèle ttr humanisé
WO2021126817A2 (fr) * 2019-12-17 2021-06-24 Aridis Pharmaceuticals, Inc. Procédés d'amélioration et d'accélération de l'expression d'anticorps
EP4125348A1 (fr) 2020-03-23 2023-02-08 Regeneron Pharmaceuticals, Inc. Animaux non humains comprenant un locus ttr humanisé affichant une mutation v30m et méthodes d'utilisation
WO2022040169A1 (fr) * 2020-08-17 2022-02-24 University Of Maryland, College Park Compositions, systèmes et procédés d'ingénierie génomique orthogonale chez la plante
KR102674574B1 (ko) * 2020-09-02 2024-06-13 한국과학기술연구원 Cas9을 위한 신규 tracrRNA 시스템
US20240002839A1 (en) * 2020-12-02 2024-01-04 Decibel Therapeutics, Inc. Crispr sam biosensor cell lines and methods of use thereof
CN113122581A (zh) * 2021-04-13 2021-07-16 上海长征医院 一种Hsf1基因敲除小鼠模型的建立及应用
WO2023108047A1 (fr) 2021-12-08 2023-06-15 Regeneron Pharmaceuticals, Inc. Modèle de maladie impliquant une myociline mutante et ses utilisations
WO2024036600A1 (fr) * 2022-08-19 2024-02-22 中国农业科学院作物科学研究所 Procédé de reproduction haploïde parthénogénétique autonome pour cellules maternelles et son utilisation dans l'élevage

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